{"id":4794,"date":"2025-10-13T16:14:23","date_gmt":"2025-10-13T13:14:23","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4794"},"modified":"2025-11-03T20:17:29","modified_gmt":"2025-11-03T17:17:29","slug":"avidity-biosciences-announces-positive-pre-bla-meeting-with-u-s-fda-for-del-zota-in-dmd44-with-a-submission-planned-for-q1-2026","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/avidity-biosciences-announces-positive-pre-bla-meeting-with-u-s-fda-for-del-zota-in-dmd44-with-a-submission-planned-for-q1-2026\/","title":{"rendered":"Avidity Biosciences, DMD44'te del-zota i\u00e7in ABD FDA ile Olumlu \u00d6n BLA Toplant\u0131s\u0131 Duyurdu ve 2026'n\u0131n 1. \u00c7eyre\u011finde Sunum Planlan\u0131yor"},"content":{"rendered":"

Avidity Biosciences, ABD G\u0131da ve \u0130la\u00e7 Dairesi (FDA) ile delpacibart zotadirsen (del-zota) adl\u0131 BLA'ya sunulmas\u0131 planlanan ila\u00e7la ilgili olarak olumlu bir \u00f6n BLA toplant\u0131s\u0131 ger\u00e7ekle\u015ftirdi\u011fini duyurdu. \u00c7\u0131\u011f\u0131r A\u00e7an Terapi unvan\u0131n\u0131 alan Del-zota, ekzon 44 atlamaya (DMD44) yatk\u0131n gen mutasyonlar\u0131 olan Duchenne kas distrofisi hastalar\u0131 i\u00e7in potansiyel bir tedavi olarak de\u011ferlendirilen ara\u015ft\u0131rma a\u015famas\u0131ndaki bir ila\u00e7t\u0131r.<\/p>\n\n\n\n

Daha fazla bilgi edin<\/strong>: Ekzon 44 Atlama Terapilerine Uygun Mutasyonlar ve Delesyonlar<\/a><\/p>\n\n\n\n

BLA g\u00f6nderim zamanlamas\u0131, FDA'nin g\u00f6nderim s\u0131ras\u0131nda kimya, \u00fcretim ve kontroller (CMC) paketini destekleyecek ek veriler almas\u0131n\u0131 sa\u011flamak amac\u0131yla, 2025 y\u0131l sonu rehberli\u011finden 2026'n\u0131n 1. \u00e7eyre\u011fine g\u00fcncellendi.<\/p>\n\n\n\n

Avidity Biosciences Ba\u015fkan\u0131 ve CEO'su Sarah Boyce, "FDA ile yapt\u0131\u011f\u0131m\u0131z son toplant\u0131 son derece i\u015f birlik\u00e7iydi ve BLA ba\u015fvurumuz i\u00e7in net bir yol sa\u011flad\u0131," dedi. "DMD i\u00e7in, ABD'deki h\u0131zland\u0131r\u0131lm\u0131\u015f onay s\u00fcreci, del-zota'yi ihtiya\u00e7 duyan ki\u015filere ula\u015ft\u0131rman\u0131n en iyi ve en h\u0131zl\u0131 yoludur. Kurumun, ba\u015fvuru s\u0131ras\u0131nda ihtiya\u00e7 duydu\u011fu CMC verileri konusunda bize net bir rehberlik sa\u011flamas\u0131ndan dolay\u0131 minnettar\u0131z ve bu uyumun ba\u015far\u0131l\u0131 bir BLA s\u00fcrecini kolayla\u015ft\u0131raca\u011f\u0131na inan\u0131yoruz. Bu program\u0131 hak etti\u011fi aciliyetle ilerletmemizi sa\u011flayan FDA b\u00fcnyesindeki \u00e7e\u015fitli birimlerden \u00f6zverili ve \u00e7al\u0131\u015fkan incelemecilere minnettar\u0131z. DMD ile ya\u015fayan erkek \u00e7ocuklar ve gen\u00e7 erkekler i\u00e7in her dakikan\u0131n \u00f6nemli oldu\u011funu biliyoruz."<\/p>\n\n\n\n

Avidity, del-zota'nin potansiyeline son derece g\u00fcveniyor ve del-zota i\u00e7in 2026'n\u0131n ilk \u00e7eyre\u011finde bir BLA ba\u015fvurusunda bulunmay\u0131 d\u00f6rt g\u00f6zle bekliyor. Bu, \u015eirketin 12 ayl\u0131k bir s\u00fcre i\u00e7inde planlad\u0131\u011f\u0131 \u00fc\u00e7 BLA ba\u015fvurusundan ilki olacak. Ayr\u0131ca Avidity, del-zota'nin k\u00fcresel olarak tam onay\u0131n\u0131 desteklemek i\u00e7in bir do\u011frulay\u0131c\u0131 \u00e7al\u0131\u015fma haz\u0131rlamaya devam ediyor.<\/p>\n\n\n\n

Devam\u0131n\u0131 oku<\/strong>:\u00a0Duchenne \u0130\u00e7in Klinik Ara\u015ft\u0131rmalar (T\u00fcm Ara\u015ft\u0131rmalar\u0131n Listesi)<\/a><\/p>","protected":false},"excerpt":{"rendered":"

Avidity Biosciences announced that the Company completed a positive pre-BLA meeting with the U.S. Food and Drug Administration (FDA) regarding its upcoming BLA submission of delpacibart zotadirsen (del-zota). Del-zota, which received Breakthrough Therapy designation, is an investigational drug being evaluated as a potential treatment for people living with Duchenne muscular dystrophy who have gene mutations […]<\/p>\n","protected":false},"author":1,"featured_media":4796,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[126,298,397,129,141,127,246,88],"class_list":["post-4794","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-avidity-biosciences","tag-del-zota","tag-delpacibart-zotadirsen","tag-dmd44","tag-exon-44","tag-exon-44-skipping","tag-exon-44-skipping-therapies","tag-fda"],"subtitle":"Avidity Biosciences, delpacibart zotadirsen'in planlanan BLA dosyas\u0131 hakk\u0131nda FDA ile ba\u015far\u0131l\u0131 bir BLA \u00f6ncesi toplant\u0131 ger\u00e7ekle\u015ftirdi\u011fini bildirdi.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4794","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=4794"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/4794\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/4796"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=4794"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=4794"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=4794"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}